Biology in the Laboratory

A Look at a Genetics Lab

What is gene therapy?

Gene therapy involves replacement of an “abnormal” disease-causing gene with a “normal” gene. The normal gene is delivered to target cells using a vector, which is usually a virus that has been genetically engineered to carry human DNA. The virus genome is altered to remove disease-causing genes and insert therapeutic genes. Target cells are infected with the virus. The virus then integrates its genetically altered material containing the human gene into the target cell, where it should produce a functional protein product.


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